NightstaRx Announces Start of Miami Gene Therapy Study

NightstaRx Ltd recently announced that the Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine, has begun enrolling and dosing subjects in a Phase II clinical trial to treat Choroideremia (CHM). This gene therapy approach uses a viral vector known as adeno-associated virus (AAV) to deliver a wild-type copy of the Rab-escort protein 1 (REP-1) gene (AAV2-REP1) into cells of the eye.

The trial, which is sponsored by Bascom Palmer Eye Institute and donors interested in retinal research, is an open label study involving a total of 6 male patients, who will each receive a single dose of AAV2-REP1, supplied by Nightstar, via a sub retinal injection.

Choroideremia is an inherited X-linked recessive disease, which inevitably causes blindness. It is caused by mutations to the CHM gene which encodes Rab-escort protein 1 and affects approximately 1 in 50,000 people. The first symptom of the condition is usually an impairment of night vision, which often occurs in early childhood. This is followed by progressive narrowing of the field of vision, as well as a decrease in the ability to see details, culminating in blindness, most commonly in late adulthood. No effective treatment currently exists.

“Gene therapy has huge potential as a treatment for many patients who are suffering from genetic causes of blindness. We have seen some very promising results from our choroideremia program and have recently expanded our pipeline to include other inherited retinal dystrophy disorders as we continue to strive to restore or maintain sight in patients suffering from these devastating blinding diseases,” said David Fellows, CEO of Nightstar.

“We are extremely excited and optimistic about the success of this gene therapy trial and are continuing to treat more patients with choroideremia with the hope that the knowledge gained will also help patients with other retinal diseases,” added Byron L. Lam, Professor at the Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine .

Nightstar is a private biopharmaceutical company focused on the development of therapies for retinal dystrophies. The company’s lead program is a retinal gene therapy for choroideremia, a rare inherited cause of blindness that affects around 1 in 50,000 people. Gene therapy has the potential to be an effective treatment for choroideremia and a range of other retinal dystrophies. The company’s lead investor, Syncona LLP, an independent subsidiary of the Wellcome Trust, is an evergreen investment company, taking an active role in identifying, supporting, and developing technologies with the potential to significantly impact the healthcare market of the future. For more information, visit www.nightstaRx.com.

Bascom Palmer Eye Institute of the University of Miami Miller School of Medicine, is ranked the nation’s best program in ophthalmology by US News & World Report. As the largest ophthalmic care, research, and educational facility in the southeastern US, more than 250,000 patients with nearly every ophthalmic condition are treated each year and more than 12,000 surgeries are performed annually. Founded in 1962, Bascom Palmer has patient care facilities in Miami, Palm Beach Gardens, Naples, and Plantation, FL. For more information, visit www.bascompalmer.org.